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A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene.
Aronson SJ, Bakker RS, Moenis S, van Dijk R, Bortolussi G, Collaud F, Shi X, Duijst S, Ten Bloemendaal L, Ronzitti G, Muro AF, Mingozzi F, Beuers U, Bosma PJ. Aronson SJ, et al. Among authors: shi x. Mol Ther Methods Clin Dev. 2020 Jun 3;18:250-258. doi: 10.1016/j.omtm.2020.06.002. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637454 Free PMC article.
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Shi X, Aronson SJ, Ten Bloemendaal L, Duijst S, Bakker RS, de Waart DR, Bortolussi G, Collaud F, Oude Elferink RP, Muro AF, Mingozzi F, Ronzitti G, Bosma PJ. Shi X, et al. Mol Ther Methods Clin Dev. 2020 Dec 3;20:287-297. doi: 10.1016/j.omtm.2020.11.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33511243 Free PMC article.
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
Shi X, Bortolussi G, Collaud F, Lebrun PR, Bloemendaal LT, Guerchet N, Rudi de Waart D, Sellier P, Duijst S, Veron P, Mingozzi F, Kishimoto TK, Ronzitti G, Bosma P, Muro AF. Shi X, et al. Mol Ther Methods Clin Dev. 2024 Oct 28;32(4):101363. doi: 10.1016/j.omtm.2024.101363. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39618425 Free PMC article.
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